Generation of an optimized lentiviral vector encoding a high-expression factor VIII transgene for gene therapy of hemophilia A
Author:
Publisher
Springer Science and Business Media LLC
Subject
Genetics,Molecular Biology,Molecular Medicine
Link
http://www.nature.com/articles/gt201276.pdf
Reference48 articles.
1. Ehrenforth S, Kreuz W, Scharrer I, Kornhuber B . Factor VIII inhibitors in haemophiliacs. Lancet 1992; 340: 253.
2. VandenDriessche T, Collen D, Chuah MK . Viral vector-mediated gene therapy for hemophilia. Curr Gene Ther 2001; 1: 301–315.
3. Roth DA, Tawa NE, O'Brien JM, Treco DA, Selden RF . Nonviral transfer of the gene encoding coagulation factor VIII in patients with severe hemophilia A. N Engl J Med 2001; 344: 1735–1742.
4. Powell JS, Ragni MV, White GC, Lusher JM, Hillman-Wiseman C, Moon TE et al. Phase 1 trial of FVIII gene transfer for severe hemophilia A using a retroviral construct administered by peripheral intravenous infusion. Blood 2003; 102: 2038–2045.
5. Berlfein J . Clinical trial update. Hemaware 2003; 8: 52–53.
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